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The FDA approves the first ever progeria drug

In this photo on February 12, 2019, Meghan Waldron walks down the street in Boston. Waldron is a student at Emerson College with progeria, one of the world’s rarest diseases. The first treatment is approved for progeria, Friday, November 20, 2020. The US Food and Drug Administration on Friday approved Zokinvy, which was shown by testing to prolong patients’ lives by an average of 2 years. (Suzanne Kreiter / The Boston Globe via AP)

TO Newsroom
UPDATED 09:19 PT – Sunday 22 November 2020

A pioneering drug against the ultra rare genetic disease known as progeria has been approved by the Food and Drug Administration (FDA). The oral capsule drug, which goes by the name Zokinvy, was given the green light on Friday.

The FDA said it is expected to reduce the risk of death for those with the fatal diagnosis. On average, it turns out to increase life expectancy by two and a half years. Research behind the treatment was largely funded by the Progeria Research Foundation (PRF).

“My hope is that the time will come when we will talk about progeria as something in the histroy books that we will have found out,” said Dr. Francis Collins, director of the National Institutes of Health. “Not just how to treat it to make it a little better, but a cure and it’s a big task.”

With this latest development, PRF has come one step closer to this goal. When the drug is widely available, about 400 people in the world with the condition, including 20 in the United States, can breathe a little easier knowing that scientists are one step closer to a cure.

Progeria, also known as Hutchinson-Gilford Progeria syndrome, has been described by the FDA as a “rare genetic disease that causes premature aging, death and has a debilitating effect on people’s lives.”

At the molecular level, it is due to an accumulation of defective progerin or progerin-like protein in cells. It causes vomiting, nausea, abdominal pain, fatigue, stroke, heart problems and more.

On average, children with progeria die around the age of 14 years. Fortunately, that will hopefully change as Zokinvy hits the market after conducting four thorough worldwide surveys over more than 10 years.

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